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Noninterventional study assessing joint health in persons with hemophilia A after switching to turoctocog alfa pegol: design of pathfinderReal
CONCLUSION: The pathfinderReal study will provide insights regarding the impact of N8-GP on joint health in persons with hemophilia A in a real-world setting.PMID:38572481 | PMC:PMC10987329 | DOI:10.1016/j.rpth.2024.102363 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 4, 2024 Category: Hematology Authors: Cihan Ay Olga Benitez-Hidalgo Gillian Gidley Maria Elisa Mancuso Davide Matino Azusa Nagao Laszlo Nemes John Waller Johannes Oldenburg Source Type: research

Immunogenicity profile of rurioctocog alfa pegol in previously treated patients with severe congenital hemophilia A
Blood Adv. 2024 Apr 2:bloodadvances.2023011780. doi: 10.1182/bloodadvances.2023011780. Online ahead of print.ABSTRACTRurioctocog alfa pegol is an extended half-life full-length recombinant factor VIII (FVIII) bound to 20 kDa polyethylene glycol (PEG) that has been shown to be well tolerated and efficacious in the treatment and prevention of bleeding events in previously treated patients with severe hemophilia A. Here, we present a comprehensive analysis of immunogenicity data collected during 6 clinical studies of rurioctocog alfa pegol including a total of 360 unique previously treated patients with severe hemophilia A. T...
Source: Adv Data - April 2, 2024 Category: Epidemiology Authors: Frank M Horling Birgit M Reipert Peter Allacher Werner Engl Luying Pan Srilatha D Tangada Source Type: research

Consensus-Based Expert Recommendations on the Management of Hemophilia A in the Gulf Region
Acta Haematol. 2024 Apr 2. doi: 10.1159/000538400. Online ahead of print.ABSTRACTBACKGROUND: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region. The aim of this manuscript is to contextualize unmet management needs, provide recommendations to optimize care, and specify requirements for the establishment of gene therapy centers in the region.SUMMARY: An expert panel was assembled comprising ten clinical hematologists from Kuwait, Oman, Saudi Arabia, and the United Arab ...
Source: Acta Haematologica - April 2, 2024 Category: Hematology Authors: Hazzaa Abdulla Alzahrani Ashraf Warsi Ali Mullah-Ali Ghazi Alotaibi Mahmoud Abu-Riash Mohammad Alshahrani Mustaqeem Siddiqui Tarek Owaidah Shahrukh Hashmi Source Type: research

Clinical Analysis and Mental Health Survey of Hemophilia Carriers: a Cross-sectional Study
ConclusionsThe detection rate of gene mutations in hemophilia mothers was 90.6%, with a median clotting factor level of 0.74 IU/mL, and 14.3% of HCs had a clotting factor level of 0.40 IU/mL or below. A history of bleeding was present in 41.2% of HCs with low clotting factor levels (less than 0.50 IU/mL). Additionally, given the fragile mental health status of HCs in China, it is critical to develop efficient strategies to improve psychological well-being. (Source: Journal of Huazhong University of Science and Technology -- Medical Sciences --)
Source: Journal of Huazhong University of Science and Technology -- Medical Sciences -- - April 2, 2024 Category: Research Source Type: research

Long-term outcomes with emicizumab in hemophilia A without inhibitors: results from the HAVEN 3 and 4 studies
CONCLUSION: With nearly 5 years of emicizumab exposure across the HAVEN 3 and 4 studies in people with HA without inhibitors, these data indicate continued bleed control with no new safety signals observed during long-term follow-up.PMID:38559572 | PMC:PMC10978536 | DOI:10.1016/j.rpth.2024.102364 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - April 1, 2024 Category: Hematology Authors: Johnny Mahlangu V íctor Jiménez-Yuste Giuliana Ventriglia Markus Niggli Simona Barlera C édric Hermans Michaela Lehle Pratima Chowdary Lyle Jew Jerzy Windyga Laurent Frenzel Christophe Schmitt Giancarlo Castaman Steven W Pipe Source Type: research

Exploring the effects of Factor Xa inhibitors on thrombin generation in people with haemophilia
Optimal management of cardiovascular disease (CVD) in people with haemophilia (PWH) is a growing issue, given the continuing improvement in life expectancy among PWH. The evolving treatment paradigms targeting higher trough levels and the advent of non-factor replacement therapies (NFRT) means much of the ‘protection’ PWH were thought to have against CVD may be lost. There is a paucity of evidence regarding the safety of using anticoagulants in PWH. We designed a study assessing the thrombin generation (TG) of PWH of different severities and treatments, compared to non-haemophilia patients receiv ing a Factor Xa (FXa) ...
Source: Thrombosis Research - March 29, 2024 Category: Hematology Authors: Caroline Dix, James D. McFadyen, Huyen A. Tran Tags: Full Length Article Source Type: research

Race and Ethnicity Reporting and Representation in Hemophilia Clinical Trials
Blood Adv. 2024 Mar 28:bloodadvances.2024012862. doi: 10.1182/bloodadvances.2024012862. Online ahead of print.ABSTRACTRacial and ethnic representativeness in clinical trials is crucial to mitigate outcomes disparities, however, diversity among hemophilia trials is unknown. The aim of this study is to examine the reporting and representation of race and ethnicity in trials of people with hemophilia (PwH). In this cross-sectional study, the clinicaltrials.gov database was queried in April 2023 for interventional clinical trials involving PwH between 2007-2022. The distribution of participants (observed) was compared with exp...
Source: Adv Data - March 28, 2024 Category: Epidemiology Authors: Stacey Fedewa Leonard A Valentino Andee Koo Lorraine Cafuir Duc Quang Tran Ana G Antun Christine L Kempton Source Type: research

Association Between Leg Muscle Thickness and Walking Test with the Haemophilia-Specific Functional Parameters
CONCLUSION: Muscle thickness and the distance of 6MWT were linked to assessment of joint health, quality of life and activity participation in PWH. Ultrasound measurement of muscle thickness and walking test appear to be useful tools for the assessment of joint health and functional status in PWH.PMID:38547026 | DOI:10.1097/PHM.0000000000002482 (Source: Health Physics)
Source: Health Physics - March 28, 2024 Category: Physics Authors: Jia-Pei Hong Jiunn-Horng Kang Chian-Her Lee Chih-Hong Lee Shu-Huey Chen Yen-Lin Liu Jia-Ruey Tsai Chen-Hua Tsai Wan-Ling Ho Mei-Mei Cheng Prangthip Charoenpong Chia-Yau Chang Source Type: research

In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia
AbstractHemophilia is a genetic disorder that is caused by mutations in coagulation factor VIII (hemophilia A) or IX (hemophilia B) genes resulting in blood clotting disorders. Despite advances in therapies, such as recombinant proteins and products with extended half-lives, the treatment of hemophilia still faces two major limitations: the short duration of therapeutic effect and production of neutralizing antibodies against clotting factors (inhibitor). To overcome these limitations, new hemophilia treatment strategies have been established such as gene therapy, bispecific antibody, and rebalancing therapy. Although thes...
Source: Molecular Diagnosis and Therapy - March 28, 2024 Category: Molecular Biology Source Type: research

Safety and efficacy of myofascial release therapy in the treatment of patients with hemophilic ankle arthropathy. Single-blind randomized clinical trial
CONCLUSION: Myofascial release therapy is a safe form of physical therapy for patients with hemophilia. Myofascial release therapy can effectively complement prophylactic pharmacological treatment in patients with hemophilic arthropathy, improving range of motion in dorsal flexion, functional capacity and gastrocnemius strength.PMID:38530214 | DOI:10.1080/09593985.2024.2334752 (Source: Physiotherapy Theory and Practice)
Source: Physiotherapy Theory and Practice - March 26, 2024 Category: Physiotherapy Authors: Elena Donoso- Úbeda Roberto Ucero-Lozano Javier Mero ño-Gallut Rub én Cuesta-Barriuso Ra úl Pérez-Llanes Source Type: research

Moderate- to vigorous-intensity physical activities for hemophilia A patients during low-dose pharmacokinetic-guided extended half-life factor VIII prophylaxis
Low-dose pharmacokinetic (PK)-guided extended half-life (EHL) factor VIII (FVIII) prophylaxis can reduce the bleeding risk in hemophilia A (HA) patients. An increase in physical activities for promoting muscul... (Source: Orphanet Journal of Rare Diseases)
Source: Orphanet Journal of Rare Diseases - March 26, 2024 Category: Internal Medicine Authors: Chonlatis Srichumpuang, Arunothai Rakmanotham, Chatphatai Moonla and Darintr Sosothikul Tags: Research Source Type: research